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Gene Replacement Therapy Suppliers & Manufacturers
95 companies found
based inGermantown, MARYLAND (USA)
Cell and gene therapies (CGTs) use modified cells or cellular matter to treat, prevent or cure serious health issues like cancer, genetic disorders, immunodeficiencies, rare diseases, and more. CGTs have shown potential to replace damaged cells with ...
based inBrisbane, CALIFORNIA (USA)
Mammoth Biosciences is harnessing the diversity of nature to power the next generation of CRISPR products. Through its discovery of novel CRISPR systems, the company is enabling the full potential of its platform to read and write the code of life. ...
Discovering novel CRISPR systems: We are addressing the current challenges of CRISPR-Cas systems and expanding the scope of use by applying our proprietary discovery platform to identify novel Cas ...
based inNew York, NEW YORK (USA)
Altucell, Inc. is a cellular engineering and biotech company backed by sound research and science focused on fulfilling a large 'unmet need' in cell, molecular and regenerative therapy for treatment of Diabetes and other autoimmune and ...
based inPleasanton, CALIFORNIA (USA)
We’re all about helping biologics and gene therapy researchers break free from tools that just don’t cut it. Unleashing problem-tackling products that make a huge difference in the real science they do every day. That’s our mantra, our promise and ...
Stunner is the only system that pulls together UV/Vis concentration, Dynamic Light Scattering (DLS) and Static Light Scattering (SLS) data from the same 2 μL sample. Dig in to your AAV to get the total capsid titer and empty/full ratio, or rack ...
based inAlpharetta, GEORGIA (US) (USA)
Clearside Biomedical is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®). The platform provides an in-office, repeatable, non-surgical procedure, offering targeted ...
We believe our platform offers the potential for safer, targeted ocular gene therapy without some of the risks of surgery and subretinal administration. Suprachoroidal administration of gene therapy could ultimately enhance access to care because it ...
based inGaithersburg, MASSACHUSETTS (USA)
In November 2023, Selecta Biosciences Inc. (NASDAQ: SELB) merged with Cartesian Therapeutics to create a publicly traded company pioneering RNA cell therapy to treat autoimmune disease. The combined company will focus on advancing Cartesian’s wholly ...
Selecta Biosciences’ immune tolerance platform, ImmTOR, is designed to give rise to antigen-specific immunity, mitigating unwanted immune ...
based inBergisch Gladbach, GERMANY
For more than 30 years, Miltenyi Biotec has played an important role in the design, development, manufacture, and integration of products that empower the advancement of biomedical research and enable cell and gene therapy. Our expertise spans ...
based inAlameda, CALIFORNIA (USA)
Since its inception in 1998, AllCells, a Discovery Life Sciences Company, has focused on advancing science in an innovative, collaborative, and client-centric manner. In our corporate video, AllCells’ Founder, Jay Tong, describes his early days in ...
AllCells’ GMP Fresh Leukopak products are collected by apheresis from peripheral blood of healthy donors. Our Leukopaks contain a high concentration of lymphocyte populations, T Cells, B Cells, NK Cells, and monocytes. AllCells maintains ...
based inBoston, MASSACHUSETTS (USA)
Neoclease is at the forefront of advancing gene editing technologies with a mission to transform healthcare by curing genetic diseases rather than just treating them. Utilizing a bespoke approach, the company develops tailored nucleases designed to ...
based inNew York, NEW YORK (USA)
We are a biopharmaceutical company developing transformational therapeutics targeting serious diseases with significant unmet medical needs. Our mission is to improve patient outcomes and save lives. To achieve our mission, we seek to capitalize on ...
Our licensed gene therapy, CO-sTiRNA™, is a dual-action STAT3 inhibitor. STAT3 is a gene that drives tumor cell growth and anti-tumor immune suppression. CO-sTiRNA™ is a highly selective and targeted gene therapy that silences the ...
based inToulouse, FRANCE
InvivoGen was founded in 1997 by scientists with technical expertise and creative excellence stemming from a longstanding history in microbiology. InvivoGen's unparalleled skill in microbial fermentation enables us to provide a wide range of ...
based inSan Carlos, CALIFORNIA (USA)
CellFE is a venture-backed company with a mission to make advanced therapies accessible to all by transforming the way gene therapies are manufactured. The company uses a microfluidics-based cell engineering platform that can be adapted universally ...
based inSan Francisco, CALIFORNIA (USA)
Tempo Bioscience, Inc (Tempo) develops human stem cells (human iPSC technology) based cell models and biosensor enabling technologies for preclinical drug discovery & development, biobanking, in vitro diagnostics, and biomarker development. Our ...
based in, DISTRICT OF COLUMBIA (USA)
Lovelace Biomedical is very much focused on gene therapy for genetic diseases testing in human clinical trials. Our ultimate aim is to give best gene therapy medicine. Lovelace Biomedical is a contract research organization that focuses on ...
Lovelace Biomedical is very much focused on gene therapy for genetic diseases testing in human clinical trials. Our ultimate aim is to give best gene therapy medicine. http://www.lovelacebiomedical.org/specialty-expertise/gene-therapy/ ...
based inRochester, MINNESOTA (USA)
Cytotheryx, Inc. is a preclinical cell therapy company focused on the development of a readily available, consistent and high-quality source of primary human hepatocytes (liver cells) and the use of those cells for research and clinical ...
At Cytotheryx, we understand that life-saving discovery in liver disease research and medicine requires a multi-faceted approach. The key to enabling this innovation is a reliable foundation. We understand this need and the opportunity to support ...
based inSanta Barbara, CALIFORNIA (USA)
Owl Biomedical, Inc. was founded in 2011 to expand the use of its exclusive cell sorting MEMS technology for cell therapy, cancer diagnostics and basic research. Operating in Santa Barbara, California, Owl Biomedical was purchased in 2013 by ...
based inMilano, ITALY
We are a clinical-stage biotechnology company pioneering the treatment of cancers with a technology platform that allows the direct delivery of immunotherapeutic payloads within the tumor microenvironment. Intrinsic resistance significantly reduces ...
Temferon™ is a lenti-virus based hematopoietic stem progenitor cell immuno-gene therapy enabling controlled and targeted interferon-α expression within cancers. Biologically delivered via engineered tumor infiltrating monocytes (Tie2 ...
based inMumbai, INDIA
Piramal Pharma Solutions (PPS) is a Contract Development and Manufacturing Organization (CDMO) offering end-to-end development and manufacturing solutions across the drug life cycle. We serve our customers through a globally integrated network of ...
based inGrove City, OHIO (USA)
Forge is developing innovative gene therapies that aim to help patients suffering from devastating rare diseases. Forge is building capacity to directly address the growing demand for gene therapy manufacturing. Forge is currently building the ...
Krabbe disease is a devastating neurodegenerative disease affecting about 1 -2.5 in 100,000 people in the U.S. and is inherited in an autosomal recessive manner. Krabbe disease is caused by loss-of-function mutations in the galactosylceramidase ...
based inElkridge, MARYLAND (USA)
Latham BioPharm Group (LBG) is a leading life sciences consulting company that works with pharmaceutical, biotech, CROs, academia, and medical device/diagnostic companies alike to solve complex business, regulatory, and product development ...